Fabry disease: The need to stratify patient populations to better understand the outcome of enzyme replacement therapy

Clinical Therapeutics

Volumn 29, Issue SUPPL. A, 2007, Pages

  Germain, Dominique P ^a  

^a ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALPHA GALACTOSIDASE;

ARTICLE; CLINICAL TRIAL; DISEASE COURSE; DRUG EFFICACY; DRUG MARKETING; DRUG SAFETY; ENZYME REPLACEMENT; FABRY DISEASE; GLOMERULOSCLEROSIS; HUMAN; KIDNEY BIOPSY; KIDNEY DISEASE; KIDNEY FUNCTION; PATHOPHYSIOLOGY; PROTEINURIA; TREATMENT OUTCOME;

ALPHA-GALACTOSIDASE; CLINICAL TRIALS AS TOPIC; FABRY DISEASE; HUMANS; ISOENZYMES; PATIENT EDUCATION AS TOPIC; TREATMENT OUTCOME;

EID: 34250214447     PISSN: 01492918     EISSN: 1879114X     Source Type: Journal    
DOI: 10.1016/S0149-2918(07)80122-6     Document Type: Article

References (11)

1. Eng C.M., Guffon N., Wilcox W.R., et al. Safety and efficacy of recombinant human alpha-galactosidase A-replacement therapy in Fabry's disease. International Collaborative Fabry Disease Study Group. N Engl J Med 345 (2001) 9-16
2. Schiffmann R., Koop J.B., Austin III H., et al. Enzyme replacement therapy in Fabry disease: A randomized controlled trial. JAMA 285 (2001) 2743-2749
3. Desnick R.J. Enzyme replacement therapy for Fabry disease: Lessons from two alpha-galactosidase A orphan products and one FDA approval. Expert Opin Biol Ther 4 (2004) 1167-1176
4. Banikazemi M., Bultas J., Waldek S., et al. Agalsidase-beta therapy for advanced Fabry disease. Fabry Disease Clinical Trial Study Group. Ann Intern Med 146 (2007) 77-86
5. European Medicines Agency Summary of Product Characteristics (2007) http://www.emea.europa.eu/humandocs/ Humans/ EPAR/fabrazyme.htm
6. Wilcox W.R., Banikazemi M., Guffon N., et al., International Fabry Disease Study Group. Long-term safety and efficacy of enzyme replacement therapy for Fabry disease. Am J Hum Genet 75 (2004) 65-74
7. Germain DP. Waldek S. Banikazemi M, et al. Sustained, long-term renal stabilization after 54 months of agalsidase beta therapyin patients with Fabry disease. J Am Soc Nephrol. In press.
8. Breunig F., Weidemann F., Strotmann J., et al. Clinical benefit of enzyme replacement therapy in Fabry disease. Kidney Int 69 (2006) 1216-1221
9. Beer M., Weidemann F., Breunig F., et al. Impact of enzyme replacement therapy on cardiac morphology and function and late enhancement in Fabry's cardiomyopathy. Am J Cardiol 97 (2006) 1515-1518
10. [published correction appears in Lancet Neurol. 2005;4:20]. Brady R.O., and Schiffmann R. Enzyme-replacement therapy for metabolic storage disorders. Lancet Neurol 3 (2004) 752-756
11. [in French]. Germain D.P. Fabry disease: From the discovery of lysosomes to enzyme replacement therapy. Med Sci (Paris) 21 (2005) 5-7